What is the Bone Marrow Failure Program?

Through our Bone Marrow Failure Program, we diagnose and treat a range of conditions that can cause bone marrow failure. We also provide many other types of care that children with bone marrow failure may need for related health problems.

Bone marrow failure means that the marrow inside the bones doesn't make enough of one or more kinds of blood cells the body needs, such as:

  • White blood cells, which fight infection
  • Red blood cells, which carry oxygen
  • Platelets, which make the blood clot and stop bleeding

The complications of bone marrow failure can be life threatening, and children with marrow failure need complex care. There are many possible causes of marrow failure. Some are inherited, meaning there is a genetic basis to their condition, while some are acquired. At Seattle Children's, we diagnose and treat both inherited bone marrow failure syndromes as well as acquired bone marrow failure.

Our Bone Marrow Failure Program is part of our larger Hematology Program, which serves children with a wide range of blood disorders. Some children with bone marrow failure have health problems that affect other body systems in addition to their blood. They may have problems with their digestion, heart, lungs, kidneys, hormones, bones, nerves or other systems. Some patients may be at higher risk for some types of cancer, too - mainly leukemia but also some solid tumors. We diagnose and treat these health problems as well, calling on specialists from different areas of healthcare depending on each child's needs.

What conditions do you treat?

We treat inherited and acquired bone marrow failure, including these conditions:

These conditions are all different and might affect the body in different ways. The symptoms of each condition depend on the cause, the types of blood cells affected and the other body systems involved. Here is a list of some of the problems that are related to bone marrow failure and some of the symptoms that can come with each of these problems.

Problem   Symptoms  
Low levels of white blood cells Higher risk of infection, getting sick more often than usual or more often than most other children
Low levels of red blood cells (anemia) Being tired, less active than usual, pale or dizzy
Low levels of platelets Bruising or bleeding easily

How do you diagnose and treat bone marrow failure?

The way we find out about what condition your child has and how to best treat them may be different depending on their symptoms or other medical issues. Most of the time, these are the steps that our team will go through to diagnose your child:

  • Take a detailed health history
  • Carefully examine your child for signs of illness
  • Do blood tests to check the level of each kind of blood cell, look at the blood cells under a microscope, and use what we see to help find out the cause of the problem
  • Remove a sample of bone marrow (called bone marrow aspiration or biopsy) to find out if there are any problems with the marrow
  • Sometimes, our team might have to do other tests. For example, we might have to get images of the inside of your child's body using a test like an ultrasound or echocardiogram. These use harmless, painless sound waves to look inside the body for other related problems that may be caused by conditions that also cause bone marrow failure.

Your child's doctor will create a treatment plan that is best suited to your child's problems and needs. In general, treatment for bone marrow failure depends on what caused the failure, in what ways and how badly your child is affected and how your child responds to treatment.

One important treatment option for some children is called a hematopoietic cell transplant. Depending on the illness that your child has, other options may also be available. Some of these treatments are:

  • Androgens
  • Growth factors for blood cells
  • Blood transfusions
  • Medicines like antithymocyte globulin (ATG) and cyclosporine

What's special about the Bone Marrow Failure Program at Seattle Children's?

Seattle Children's works with the Fred Hutchinson Cancer Research Center and Seattle Cancer Care Alliance (SCCA) to offer hematopoietic stem cell transplants. We also work with these partners to do research on bone marrow failure so we can understand the causes and develop better treatments. The Hutchinson Center is a leader in transplants. They have extensive experience in the kind of transplants that many patients with bone marrow failure need (reduced-intensity conditioning regimens). SCCA has developed special transplant treatments made for patients with inherited bone marrow failure syndromes.

Seattle Children's is also working with hematologists at the University of Washington to improve the care of adults with inherited marrow failure syndromes.

Our partner, the University of Washington is home to the Severe Chronic Neutropenia International Registry (SCNIR), which helps track people who have a condition that results in having a shortage of white blood cells (white blood cell deficiency) that can be caused by bone marrow failure. Registries like this collect data to help experts learn more about diseases and their treatment.

We are actively involved in family groups and other efforts to help find out more about treating bone marrow failure and to provide support for patients and families, such as the Shwachman-Diamond Syndrome Foundation, Fanconi Anemia Research Fund, National Neutropenia Network and Daniella Maria Arturi Foundation.

Who needs the Bone Marrow Failure Program?

This program is for children:

  • Who might have bone marrow failure and who need a diagnosis
  • With known bone marrow failure who need treatment

At Seattle Children's, we work with many children and families from around the Northwest and beyond. Whether you are as close as Seattle or will be traveling from far away to come to Seattle Children's, we can help you with things like getting ready for your child's clinic visit or hospital stay, transportation, neighborhood services, billing and financial assistance, places to stay and more.